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Recombinant Factor VIII

efanesoctocog alfa (BIVV001) for Hemophilia A (XTEND-Kids Trial)

Phase 3
Waitlist Available
Research Sponsored by Bioverativ, a Sanofi company
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Weight above or equal to 10 kg.
Participant must be younger than 12 years of age, at the time of signing the informed consent
Timeline
Screening 3 weeks
Treatment Varies
Follow Up pre-dose, 0.25, 3, 24, 72, and 168 hours post-dose on day 1
Awards & highlights

XTEND-Kids Trial Summary

This trial is testing a new medication, BIVV001, for safety in children with hemophilia A who have been treated before. They will also look at how well it works as a preventative treatment and for treating bleeding episodes, how much of the medication is needed, and any side effects.

XTEND-Kids Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

XTEND-Kids Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~pre-dose, 0.25, 3, 24, 72, and 168 hours post-dose on day 1
This trial's timeline: 3 weeks for screening, Varies for treatment, and pre-dose, 0.25, 3, 24, 72, and 168 hours post-dose on day 1 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With Neutralising Antibodies (Development of Inhibitors) Directed Against Factor VIII
Secondary outcome measures
Annualized Bleeding Rate (ABR): For Treated Bleeds
Annualized Bleeding Rate by Location of Bleed (Joint, Muscle, Internal and Skin/Mucosa)
Annualized Bleeding Rate by Type of Bleed (Spontaneous, Traumatic and Unknown Type)
+44 more

Side effects data

From 2022 Phase 3 trial • 159 Patients • NCT04161495
20%
Headache
16%
Arthralgia
6%
Back Pain
6%
Fall
4%
Fatigue
1%
Covid-19 Pneumonia
1%
Basal Cell Carcinoma
1%
Pancreatic Carcinoma Metastatic
1%
Cubital Tunnel Syndrome
1%
Status Epilepticus
1%
Ulnar Tunnel Syndrome
1%
Angina Pectoris
1%
Arthropathy
1%
Haemophilic Arthropathy
1%
Traumatic Haemorrhage
1%
Central Venous Catheter Removal
1%
Mobility Decreased
1%
Blood Glucose Increased
1%
Cd4 Lymphocytes Decreased
1%
Combined Tibia-Fibula Fracture
1%
Device Breakage
100%
80%
60%
40%
20%
0%
Study treatment Arm
BIVV001 (Efanesoctocog Alfa): All Participants
Arm A: Prophylaxis
Arm B: Prophylaxis
Arm B: On-demand

XTEND-Kids Trial Design

2Treatment groups
Experimental Treatment
Group I: BIVV001: Participants aged <6 YearsExperimental Treatment1 Intervention
Participants aged less than (<) 6 years received BIVV001 at a dose of 50 international units per kilogram (IU/kg) intravenous (IV) injection once-weekly (QW) prophylaxis for 52 weeks.
Group II: BIVV001: Participants aged 6 to <12 YearsExperimental Treatment1 Intervention
Participants aged 6 to <12 years received BIVV001 at a dose of 50 IU/kg IV injection QW prophylaxis for 52 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
efanesoctocog alfa (BIVV001)
2021
Completed Phase 3
~240

Find a Location

Who is running the clinical trial?

Bioverativ, a Sanofi companyLead Sponsor
17 Previous Clinical Trials
962 Total Patients Enrolled
6 Trials studying Hemophilia A
593 Patients Enrolled for Hemophilia A
Clinical Sciences & OperationsStudy DirectorSanofi
857 Previous Clinical Trials
2,018,881 Total Patients Enrolled
9 Trials studying Hemophilia A
1,124 Patients Enrolled for Hemophilia A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the precedents for this research?

"Bioverativ, a subsidiary of Sanofi, first began to study efanesoctocog alfa (BIVV001) in 2021. After the initial trial with 75 participants in that same year, Phase 3 drug approval was given. As of now, there are three active studies being conducted for efanesoctocog alfa (BIVV001), located in 41 different cities and 23 countries around the world."

Answered by AI

Are there any observed side effects of efanesoctocog alfa (BIVV001)?

"There is a significant body of evidence both for and against the efficacy of efanesoctocog alfa (BIVV001), so it received a score of 3."

Answered by AI

What are the results of efanesoctocog alfa (BIVV001) clinical trials thus far?

"In 2021, efanesoctocog alfa (BIVV001) was first trialled at Investigational Site Number: 0560001. Since then, there have been 3 completed studies with many more ongoing clinical trials, some of which are based in Greenville, North carolina."

Answered by AI

How many different sites are participating in this research?

"There are 26 different hospitals and clinics running this clinical trial at the moment. They are located in Greenville, Ottawa and Pittsburgh as well as 23 other places. If you want to minimize your travel requirements, try and select a location that is close to you."

Answered by AI

How many individuals are under observation in this clinical trial?

"Although this study was posted on February 19th, 2021 and updated recently on May 10th, 2022, it is not currently recruiting patients. If you are interested in participating in other clinical trials, there are 91 studies searching for patients with hemophilia a and 3 efanesoctocog alfa (BIVV001) studies that still need participants."

Answered by AI

Are new participants being enrolled in this clinical trial at this time?

"Unfortunately, this clinical trial is not looking for any more participants at the moment. The original posting was on February 19th, 2021 and there have been edits as recently as May 10th, 2022. However, if you are seeking other studies, 91 trials for patients with hemophilia A and 3 trials for efanesoctocog alfa (BIVV001) are currently admitting new members."

Answered by AI
~18 spots leftby Mar 2025